Post on 14-Jul-2020
Health Canada’s role - Drugs for rare diseases
Megan Bettle, Health Canada
CADTH symposium
April 15, 2019
Challenges – Drugs for Rare Disease Development and Patient Access
Challenging to develop:
Technical challenges (e.g. lack of basic science, small patient populations, need for non-standard trials)
Regulatory challenges (e.g. different international approaches)
Regulation of companion diagnostics
Business challenges (e.g. range in profitability may be significant for small populations, some lack business incentive to develop)
Once developed and authorized for market:
Can be costly and may be difficult to implement, especially outside of specialist centres.
May be inequitable access - public and private drug plans may make different reimbursement decisions; funding recommendations may have smaller scope than HC-authorized indications based on cost-effectiveness assessment
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Health Canada as Regulator
• Under the Food and Drugs Act,
Health Canada regulates,
evaluates and monitors the
safety, efficacy, and quality of
therapeutic products
• Includes product oversight at all
stages of life cycle, from early
testing in clinical trials, to post-
market surveillance of adverse
drug reactions, and compliance
monitoring
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Drug Submission Review
• Sponsors must provide sufficient evidence of the product's safety, efficacy and quality
for assessment, including
– Manufacturing details and controls to ensure consistent quality
– Pre-clinical data to establish safety
– Clinical data to support proposed indication, including “substantial evidence of effectiveness”
– Proposed labels – claims, instructions for use
– Assessment of possibility for name confusion
• Health Canada then reviews the evidence and decides whether the benefits of the
product outweigh the risks, and whether the risks can be managed
• Health Canada continues to receive supplementary submissions after first Notice of
Compliance granted, for any significant change, including:
– New indications
– Manufacturing changes
• ~ 30% of the new drugs authorized in 2018 are considered orphan drugs in other
jurisdictions
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Pathways to Drug Access
Regulatory authorization:
New Drug Submission/Supplementary New Drug Submission – review for safety, quality efficacy
• Target review time – 300 days [similar review for generics = 180 days]
Priority review policy
• Unmet medical need, serious, life-threatening or severely debilitating disease
• Requirement for “substantial” evidence of effectiveness
• Target review time – 180 days [In 2018, 4 drugs for rare diseases were reviewed under this pathway]
Notice of Compliance with Conditions policy
• Unmet medical need, serious, life-threatening or severely debilitating disease
• “Promising” evidence of effectiveness
• Target review time – 200 days [In 2018, 1 drug for a rare disease was reviewed under this pathway]
• Manufacturer agrees to conduct confirmatory studies
Other Access:
Special Access Programme
• Access to drugs which are not authorized in Canada, when other treatments exhausted or not possible
• Initiated by prescriber for individual patients
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Post-market Monitoring and Surveillance
• Once a product has been marketed in Canada, there are ongoing
obligations for sponsors, including:
– Reporting of adverse drug reactions (ADRs)
– Preparing annual summary reports on safety
– Managing any changes in the benefit-risk profile
• Health Canada conducts ongoing analysis of ADRs, environmental
scanning, ongoing assessment of potential safety issues
– As needed, issues risk communications to inform Canadians and prescribers of
changes
– Updates product labelling to ensure accuracy and to support safe use
– If benefit-risk no longer favourable, products can be withdrawn from market
– Relies on many sources of data, including clinical trials, individual case reports,
and real world evidence
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Transforming how we regulate
Objective: An agile regulatory system that supports better access to therapeutic
products based on healthcare system needs
Expanded collaboration with
health partners
• Alignment of the Health
Technology Assessment
(CADTH) Review with Health
Canada Review
• Implementing a Mechanism for
Early Parallel Scientific Advice
• Use of Foreign
Reviews/Decisions
• International Collaboration and
Work Sharing in Reviews
More timely access to drugs and
devices
• Expansion of Priority Review
Pathways
• Improving Access to Biosimilars
and Biologics
• Improving Access to Generic Drugs
• Building Better Access to Digital
Health Technologies
• Pre-Submission Scientific Advice
for Medical Devices
• Special Access Programme (SAP)
Renewal and Block Release
Enhanced use of real world
evidence
• Leveraging Data for
Assessing Drug Safety and
Effectiveness
• Strengthening the use of
real world evidence and
regulations for medical
devices
Modern and flexible operations
Updated System Infrastructure
Appropriate cost recovery framework
Public Release of Clinical Information
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R2D2 – Some Accomplishments to Date
Expanded collaboration
Established mechanisms for aligned regulatory and health technology assessment reviews, with potential to save overall time to market
19 aligned reviews completed, 9 ongoing, ~75% are new drugs
Created processes to share submission review with other regulators
2 reviews completed, more ongoing (partnering with Australia)
More timely availability of drugs and devices
Draft guidance document describing revised criteria for accelerated review, which can include drugs for rare diseases
Ongoing operational updates to the Special Access Program to reduce burden on prescribers
Expanded Use of Real World Evidence
Providing guidance on quality of evidence needed to support regulatory decision-making and encouraging submissions using RWE
Modern and Flexible Operations
Final publication of regulations supporting release of clinical information from drug submissions
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What’s Next?
Interim report on implementation of national pharmacare:
“The Council also recognizes the unique challenges of funding and accessing expensive
drugs for rare diseases. Special consideration is needed to determine how to address these
challenges to ensure a nationally consistent approach for these medications.”
Budget 2019 commitments:
• Creation of a “Canadian drug agency” - assessment, pricing negotiation, national
formulary
• National strategy for EDRDs – up to $1B over 2 years in 2022-23, $500M ongoing
Creating flexibility in the regulatory system to support innovation in bio-sciences:
• Budget Implementation Act includes new pathway for “advanced therapeutic products”
– Creation of “regulatory sandboxes” to support innovative products that don’t fit well
into existing regulatory frameworks
• Modernizing clinical trial regulation
– To become more risk-based and agile
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